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A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis

Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.

Treatment Protocol of the First International Study of Langerhans Cell Histiocytosis in adults

To define and implement an uniform initial evaluation and stratification on adult patients with LCH, to uniform the treatment of adult patients with LCH and to improve treatment results with respect to survival, therapy response, prevention of…

Convalescent Antibody-Mediated Treatment of COVID-19 Infections in Patients with B-cell dysfunction, a Randomized Trial (COVID-Compromise Study).

There have been reports of a more serious course of COVID-19 infections in patients with a reduced immune system, such as patients with congenital immune disorders, (haematological) malignancies or patients taking medications that suppress the…

Multicenter, open*label, phase II study in patients with monoclonal
gammopathy of unknown significance (MGUS) and anti Myelin Associated
Gycoproteine (MAG) Neuropathy and Zanubrutinib Treatment * MAGNAZ trial

Main objective:To improve the functional neurological outcome of patients, as measured on the Raschbuilt Overall Disability Scale (RODS) for inflammatory neuropathies (iRODS) with Zanubrutinib in combination with standard treatment with Rituximab or…

A Phase 2 Study of Daratumumab Subcutaneous (Dara-SC) Administration in Combination with Carfilzomib and Dexamethasone (DKd) Compared with Carfilzomib and Dexamethasone (Kd) in Participants with Multiple Myeloma who have been Previously Treated with Daratumumab to Evaluate Daratumumab Retreatment

The purpose of this study is to compare the efficacy (rate of very good partial response [VGPR] or better as best response as defined by the International Myeloma Working Group [IMWG] criteria) of daratumumab subcutaneous (Dara-SC) in combination…

A RANDOMIZED, 2-ARM, PHASE 3 STUDY OF ELRANATAMAB (PF-06863135) VERSUS LENALIDOMIDE IN PATIENTS WITH NEWLY DIAGNOSED MULTIPLE MYELOMA AFTER UNDERGOING AUTOLOGOUS STEM-CELL TRANSPLANTATION

This study has been transitioned to CTIS with ID 2023-508897-27-00 check the CTIS register for the current data. Primary:• To compare the efficacy of elranatamab vs lenalidomide Secondary:- To compare the efficacy of elranatamab vs lenalidomide- To…

A Multicentre, Randomised, Double-blind, Parallel-group, Placebo controlled, 24-week Phase 3 Study with an Open-label Extension to Evaluate the Efficacy and Safety of Benralizumab in Patients with Hypereosinophilic Syndrome (HES)

This study has been transitioned to CTIS with ID 2023-510455-28-00 check the CTIS register for the current data. This study is being carried out to investigate the treatment with benralizumab in patients with active HES.The purpose of this study is…

Phase 3, Two-Stage, Randomized, Multicenter, Open-label Study Comparing Iberdomide, Daratumumab and Dexamethasone (IberDd) versus Daratumumab, Bortezomib, and Dexamethasone (DVd) in Subjects with Relapsed or Refractory Multiple Myeloma (RRMM)

This study has been transitioned to CTIS with ID 2024-510800-35-00 check the CTIS register for the current data. Primary objective:To compare the efficacy of iberdomide, daratumumab and dexamethasone (IberDd) to that of daratumumab, bortezomib and…

A Phase 2 Open-Label, Multicenter Clinical Study of the Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Profiles of CGT9486 as a Single Agent in Patients With Advanced Systemic Mastocytosis

This study has been transitioned to CTIS with ID 2024-511407-42-00 check the CTIS register for the current data. Part 1: dose optimizationTo identify a clinically active and tolerable systemic exposure range of bezuclastinib in subjects with…